FDA Fast Tracks Gene Therapy for X-Linked Retinitis Pigmentosa

The Company is currently conducting a Phase 1/2 clinical trial in adult and pediatric XLRP patients with RPGR-ORF 15 mutations

The Food and Drug Administration (FDA) has granted Fast Track designation to AAV-RPGR (MeiraGTx Limited), a gene therapy candidate intended for the treatment of X-linked retinitis pigmentosa (XLRP) due to defects in the retinitis pigmentosa GTPase regulator (RPGR) gene.

AAV-RPGR is being developed to treat the most common form of XLRP, which is caused by mutations in the eye specific form of the RPGR gene, RPGR-ORF 15. The Company is currently conducting a Phase 1/2 clinical trial in adult and pediatric XLRP patients with RPGR-ORF 15 mutations.

Related Articles

The FDA had previously granted AAV-RPGR Orphan Drug designation. XLRP represents the most severe form of retinitis pigmentosa (RP), an inherited retinal disease characterized by progressive retinal degeneration and vision loss that ends in complete blindness. There are currently no approved treatments for RP.

“This Fast Track designation is an important milestone for both patients living with XLRP and MeiraGTx, allowing our team to communicate closely and often with the FDA as we work to bring a much-needed therapy to patients,” said Alexandria Forbes, PhD, president and CEO of MeiraGTx.